PBI Manufacture Core

PBI制造核心

基本信息

批准号：
7812200
负责人：
Kenneth Hadlock
金额：
$ 20.19万
依托单位：
UNIVERSITY OF CALIFORNIA, SAN FRANCISCO
依托单位国家：
美国
项目类别：
财政年份：
资助国家：
美国
起止时间：
至
项目状态：
未结题

来源：
https://reporter.nih.gov/project-details/7812200
关键词：
AIDS Dementia Complex Anabolism Animal Model Animals Antineoplastic Agents Archives Biological Biological Assay Biotechnology Clinical Clinical Investigator Clinical Trials Commit Comparative Study Consult Consultations Contracts Development Disease Documentation Drug Formulations Drug Stability Ensure GMP lots Generations HIV Human In Vitro Investigational Drugs Investigational New Drug Application Joints Label Lead Lymphoma Macaca mulatta Maintenance Malignant Neoplasms Modeling Monitoring Clinical Trials Pamphlets Pharmaceutical Preparations Pharmacologic Substance Phase Phase I Clinical Trials Phase I/II Trial Polyamines Preparation Production Program Research Project Grants Quality Control Recording of previous events Records Regulatory Affairs Research Personnel Resistance Rights Running SIV SIV encephalitis Sterility Supervision Syndrome Testing Time Toxicology United States Food and Drug Administration United States National Institutes of Health Update Vial device Work Writing analog base chemotherapy clinical lot cost drug development experience human subject inhibitor/antagonist macrophage manufacturing process nervous system disorder programs quality assurance research clinical testing small molecule stability testing sterility testing

项目摘要

This program project envisions running two small proof of concept phase I clinical trials in human subjects. To facilitate the work of this program project grant, Pathologica will be responsible for all aspects of the manufacture, quality control, and quality assurance required to produce an investigational new drug. This component includes, the manufacture of the compound(s), packaging of the drug(s), stability testing, production of clinical investigator brochures, and filing of the INDs for both trials. The first trial is envisioned to involve the polyamine biosynthesis inhibitor PA-001 in the setting of HIV associated dementia and related syndromes. PA-001 has shown excellent activity against SIV encephalitis in rhesus macaque model (see Project 2: Animal model studies for more details). The compound for the second trial will be chosen based on the results of the in vitro and animal studies performed during the first two years of the program project, but will also be a polyamine biosynthesis inhibitor. Pathologica LLC is fortunate to have rights to evaluate and commercialize many PBIs including the polyamine analog CG47, and the PBI manufacture core will ensure that sufficient quantity and quality of these polyamine analogs will be available for comparative studies with PA-001 as described more fully in Projects 1 and 2 of this program project grant. To provide the drugs Pathologica will be assisted by Dr Fredrick Johnson, who has more than 20 years of experience in the manufacture and quality assurance testing of both small molecule and biological pharmaceutical products.

该计划项目设想在人类受试者中进行两项小型概念验证一期临床试验。为了促进该计划项目拨款的工作，Pathologica 将负责该计划的所有方面生产研究性新药所需的生产、质量控制和质量保证。这组成部分包括化合物的制造、药物的包装、稳定性测试、制作临床研究者手册，并为两项试验提交 IND。预计首次审判将多胺生物合成抑制剂 PA-001 应用于治疗 HIV 相关痴呆症和相关疾病综合症。 PA-001 在恒河猴模型中表现出优异的抗 SIV 脑炎活性（参见项目 2：动物模型研究（了解更多详情）。第二次试验的化合物将根据根据该计划项目头两年进行的体外和动物研究的结果，但也将是多胺生物合成抑制剂。 Pathologica LLC 有幸拥有评估权并将包括多胺类似物CG47在内的多种PBI商业化，PBI制造核心将确保这些多胺类似物的数量和质量足以用于比较使用 PA-001 进行研究，如本计划项目拨款的项目 1 和 2 中更详细地描述。提供病理学药物将由 Fredrick Johnson 博士协助，他在病理学领域拥有 20 多年的经验小分子和生物制药产品的制造和质量保证测试。